News

This page will compile news and announcements about the PaVe-GT program, as well as highlight the outreach activities of project staff including, presentations at relevant workshops, meetings and conferences.

2022
NCATS Receives FDA Orphan Drug Designation to Treat Propionic Acidemia
The U.S. Food and Drug Administration (FDA) has granted the National Institutes of Health (NIH)’s National Center for Advancing Translational Sciences (NCATS) an orphan drug designation for AAV9-hPCCA (NCATS-BL0746), an investigational gene therapy for the treatment of propionic acidemia resulting…
2020
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